Epidermolysis Bullosa (RDEB)
Topical QR-313 in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Due to Mutation(s) in Exon 73 of the COL7A1gene (WINGS)
A First in Human, Double-blind, Randomized, Intra-subject Placebo-controlled, Multiple Dose Study of QR-313 Evaluating Safety, Proof of Mechanism, Preliminary Efficacy and Systemic Exposure in Subjects With RDEB Due to Mutation(s) in Exon 73 of the COL7A1 Gene
Four medical centers in CA, CO, MN, and OH are recruiting participants who have specific types of Recessive Dystrophic Epidermolysis Bullosa to test a new, topical treatment. The treatment aims to slow progression of EB wounds through a drug that binds to and stops the cellular messaging system that causes inflammation.
The actual drug and a placebo will be applied 2 to 3x per week to two wound areas, as assigned by the study, for 4 weeks. Home and site visits will follow for regular assessments up to 8 weeks post the last dose.
- Be 6 years of age or older
- Have at least 1 target wound area
- Have life expectancy > 6 months
THE STUDY INVOLVES:
- Screening before the treatment.
- Visit to the study center for assignment of the drug and/or placebo to the targeted wounds.
- For 4 weeks, the medicines will be applied to the assigned wounds 2 to 3 times per week.
- Home and site visits will follow for regular assessments up to 8 weeks post the last dose.
LOCATIONS AND CONTACTS:
The study locations include:
- Stanford University School of Medicine, LPCH in Palo Alto, CA
Contact: Kunju Sridhar | 650-721-4902 | [email protected]
- Children’s Hospital Colorado in Aurora, CO
Contact: Kathleen Peoples | 720-777-4708 | [email protected]
- Journey Clinic, Center for Pediatric Blood and Marrow Transplantation in Minneapolis, MN
Contact: Christen Ebens, MD | 612-626-8094 | [email protected]
- Cincinnati Children’s Hospital in Cincinnati, OH
Contact: Bret Augsburger | 513-803-9009 | [email protected]
Or go online: https://clinicaltrials.gov/ct2/show/NCT03605069